Gene therapy for treating Mucopolysaccharidosis type II
US11819539B2 · kind B2 · utility
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30Claims
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Key dates
| Filing date | Sep 21, 2018 |
| Grant date | Nov 21, 2023 |
| Priority date | — |
| Expiry date | Oct 27, 2039 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14143
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDS gene and two or more immunosuppressants is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.